AMT Submits its Lead Product Glybera(R) Application for Marketing Authorisation Application to EMA

By Amsterdam Molecular Therapeutics B.v, PRNE
Sunday, January 10, 2010

AMSTERDAM, January 11 - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field
of human gene therapy, announced today that the Marketing Authorisation
Application (MAA) for Glybera(R), AMT's proprietary lead product, for
lipoprotein lipase deficient patients, has been submitted to the European
Medicines Agency (EMA, formerly known as EMEA).

AMT has conducted two clinical studies for lipoprotein lipase deficiency
(LPLD) in Europe and Canada and long term follow-up from these is ongoing, as
is a further study in Canada. In these three studies Glybera(R) has shown a
sizeable decrease in the incidence of pancreatitis, or acute inflammation of
the pancreas, the most debilitating complication of LPLD. Moreover, these
studies also indicate that Glybera(R) has an excellent safety profile.

"We are delighted to have submitted the Glybera(R) dossier to EMA. We
believe that AMT will be the first to successfully develop a gene therapy for
a disease caused by a genetic defect," said Jörn Aldag, Chief Executive
Officer of AMT. "Importantly, Glybera(R) also validates AMT's
adeno-associated viral (AAV) vector delivery platform, which can be used to
deliver other gene therapy products for other indications."

The MAA for Glybera(R) is now in the validation stage, and the formal
review process is expected to begin later this month. The MAA for Glybera(R)
will be reviewed via the centralised procedure which is the standard route
for all advanced therapies.

About the Disease

LPLD is a seriously debilitating, and potentially lethal, orphan disease,
for which no approved therapy exists today. The disease is caused by
mutations in the LPL gene, resulting in highly decreased or absent activity
of LPL protein in patients. This protein is needed in order to break down
large fat-carrying particles that circulate in the blood after each meal.
When such particles, called chylomicrons, accumulate in the blood, they may
obstruct small blood vessels, which in turn can lead to pancreatitis.
Recurrent pancreatitis in LPLD patients can result in difficult-to-treat
diabetes. LPLD is associated with significant morbidity and mortality.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in the
development of human gene based therapies. Using AAV as the delivery vehicle
of choice for therapeutic genes, the company has been able to design and
validate what is probably the first stable and scalable AAV production
platform. This safe and efficacious proprietary platform offers a unique
manufacturing capability which can be applied to a large number of rare
(orphan) diseases that are caused by one faulty gene. Currently, AMT has a
product pipeline with several AAV-based gene therapy products in LPLD,
Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson's Disease at
different stages of research or development.

AMT will be presenting at the Biotech Showcase Conference, Marines
Memorial Club and Hotel, 609 Sutter Street, San Francisco at 1600 (PCT) on
Tuesday January 12, 2010.

Certain statements in this press release are "forward-looking statements"
including those that refer to management's plans and expectations for future
operations, prospects and financial condition. Words such as "strategy,"
"expects," "plans," "anticipates," "believes," "will," "continues,"
"estimates," "intends," "projects," "goals," "targets" and other words of
similar meaning are intended to identify such forward-looking statements.
Such statements are based on the current expectations of the management of
Amsterdam Molecular Therapeutics only. Undue reliance should not be placed on
these statements because, by their nature, they are subject to known and
unknown risks and can be affected by factors that are beyond the control of
AMT. Actual results could differ materially from current expectations due to
a number of factors and uncertainties affecting AMT's business, including,
but not limited to, the timely commencement and success of AMT's clinical
trials and research endeavors, delays in receiving U.S. Food and Drug
Administration or other regulatory approvals (i.e. EMA, Health Canada),
market acceptance of AMT's products, effectiveness of AMT's marketing and
sales efforts, development of competing therapies and/or technologies, the
terms of any future strategic alliances, the need for additional capital, the
inability to obtain, or meet, conditions imposed for required governmental
and regulatory approvals and consents. AMT expressly disclaims any intent or
obligation to update these forward-looking statements except as required by
law. For a more detailed description of the risk factors and uncertainties
affecting AMT, refer to the prospectus of AMT's initial public offering on
June 20, 2007, and AMT's public announcements made from time to time.


For more information: Jörn Aldag, Chief Executive Officer, Tel +31-(0)-20-566-7394, j.aldag at

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